A phase IIIb, Multicenter, Open-Label, Single-Arm Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Subcutaneous Emicizumab in Patients From Birth to 12 Months of Age with Hemophilia A without Inhibitors - MO41787

F. Hoffmann-La Roche AG0 个研究点目标入组 65 人开始时间: 2024年2月1日最近更新: 2024年7月22日

研究概览

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•1. No history of documented FVIII inhibitor (i.e., < 0.6 BU/mL), FVIII drug-elimination half-life < 6 hours, or FVIII recovery < 66%, 2. Mandatory receipt of vitamin K prophylaxis according to local standard practice, 3. Diagnosis of severe congenital hemophilia A (intrinsic FVIII level < 1%), 4. A negative test for FVIII inhibitor (i.e., < 0.6 Bethesda units [BU]/mL) locally assessed during the 2-week screening period for all patients, 5. Previously untreated patients (PUPs) or minimally treated patient (MTPs) (i.e., up to 5 days of exposure with hemophilia-related treatments, such as plasma-derived FVIII, recombinant FVIII, fresh frozen plasma, cryoprecipitate, or whole blood products), 6. Documentation of the details of the hemophilia-related treatments received since birth and documentation of the details of the bleeding episodes since birth

•1. Inherited or acquired bleeding disorder other than severe hemophilia A, 2. Use of systemic immunomodulators (e.g., interferon) at enrollment or planned use during the study, 3. Current active severe bleed, such as intracranial hemorrhage (ICH), 4. History of clinically significant hypersensitivity associated with monoclonal antibody therapies or components of the emicizumab injection, 5. Patients who are at high risk for thrombotic microangiopathy (TMA) (e.g., have a previous medical or family history of TMA, such as thrombotic thrombocytopenic purpura, atypical hemolytic uremic syndrome) in the investigator's judgment, 6. Previous or current treatment for thromboembolic disease or signs of thromboembolic disease

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